Base editing treats spinal muscular atrophy in mice
A one-time genome-editing treatment restores motor function and extends lifespan in an animal model of a neuromuscular disease that is the leading genetic cause of infant mortality.
March 30, 2023
This work was supported in part by the National Institutes of Health, the Bill and Melinda Gates Foundation, the Howard Hughes Medical Institute, and the Friedreich’s Ataxia Research Alliance (FARA).
Arbab M, Matuszek Z et al. Base editing rescue of spinal muscular atrophy in cells and in mice. Science. Online March 30, 2023. DOI:10.1126/science.adg6518